WO2004010106A3 - Methodes destinees a identifier des petites molecules modulant la terminaison de traduction prematuree et la degradation de l'arnm induite par mutation non-sens - Google Patents

Methodes destinees a identifier des petites molecules modulant la terminaison de traduction prematuree et la degradation de l'arnm induite par mutation non-sens Download PDF

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Publication number
WO2004010106A3
WO2004010106A3 PCT/US2003/023075 US0323075W WO2004010106A3 WO 2004010106 A3 WO2004010106 A3 WO 2004010106A3 US 0323075 W US0323075 W US 0323075W WO 2004010106 A3 WO2004010106 A3 WO 2004010106A3
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WO
WIPO (PCT)
Prior art keywords
methods
rna
compounds
translation termination
present
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Ceased
Application number
PCT/US2003/023075
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English (en)
Other versions
WO2004010106A2 (fr
Inventor
Ellen M Welch
Neil Gregory Almstead
Robert F Rando
Mathew C Pellegrini
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PTC Therapeutics Inc
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PTC Therapeutics Inc
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Publication date
Application filed by PTC Therapeutics Inc filed Critical PTC Therapeutics Inc
Priority to AU2003252136A priority Critical patent/AU2003252136A1/en
Priority to CA002493808A priority patent/CA2493808A1/fr
Priority to US10/521,775 priority patent/US7927791B2/en
Priority to EP03765998A priority patent/EP1543157A4/fr
Publication of WO2004010106A2 publication Critical patent/WO2004010106A2/fr
Publication of WO2004010106A3 publication Critical patent/WO2004010106A3/fr
Anticipated expiration legal-status Critical
Ceased legal-status Critical Current

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    • B—PERFORMING OPERATIONS; TRANSPORTING
    • B82—NANOTECHNOLOGY
    • B82Y—SPECIFIC USES OR APPLICATIONS OF NANOSTRUCTURES; MEASUREMENT OR ANALYSIS OF NANOSTRUCTURES; MANUFACTURE OR TREATMENT OF NANOSTRUCTURES
    • B82Y30/00—Nanotechnology for materials or surface science, e.g. nanocomposites
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N33/00—Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
    • G01N33/48—Biological material, e.g. blood, urine; Haemocytometers
    • G01N33/50—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
    • G01N33/53—Immunoassay; Biospecific binding assay; Materials therefor
    • G01N33/5308—Immunoassay; Biospecific binding assay; Materials therefor for analytes not provided for elsewhere, e.g. nucleic acids, uric acid, worms, mites
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2500/00—Screening for compounds of potential therapeutic value
    • G01N2500/04—Screening involving studying the effect of compounds C directly on molecule A (e.g. C are potential ligands for a receptor A, or potential substrates for an enzyme A)

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  • Engineering & Computer Science (AREA)
  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Molecular Biology (AREA)
  • Immunology (AREA)
  • Nanotechnology (AREA)
  • Biomedical Technology (AREA)
  • General Physics & Mathematics (AREA)
  • Physics & Mathematics (AREA)
  • Hematology (AREA)
  • Urology & Nephrology (AREA)
  • Medicinal Chemistry (AREA)
  • Biotechnology (AREA)
  • Microbiology (AREA)
  • Cell Biology (AREA)
  • Analytical Chemistry (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Food Science & Technology (AREA)
  • Pathology (AREA)
  • Composite Materials (AREA)
  • Condensed Matter Physics & Semiconductors (AREA)
  • Materials Engineering (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Tropical Medicine & Parasitology (AREA)
  • Investigating Or Analysing Biological Materials (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)

Abstract

La présente invention concerne une méthode de criblage et d'identification de composés modulant la terminaison de traduction prématurée et/ou la dégradation de l'acide ribonucléique messager (ARNm) induite par mutation non-sens par interaction avec un acide ribonucléique (ARN) cible présélectionné. Plus particulièrement, la présente invention concerne l'identification de composés se liant à des régions de l'ARN ribosomique (ARNr) 28S et d'analogues correspondants. On utilise avantageusement des analyses de liaison non compétitive directe pour cribler des bibliothèques de composés en vue d'identifier les composés qui se lient sélectivement à un ARN cible présélectionné. La liaison des molécules d'ARN cible à un composé particulier est détectée au moyen d'une méthode physique permettant de mesurer la propriété physique modifiée de l'ARN cible lié à un composé. On détermine également la structure du composé fixé sur l'ARN marqué. Les méthodes utilisées dépendent en partie de la nature de la bibliothèque criblée. Les méthodes de la présente invention fournissent une analyse sensible et simple pour le criblage à vitesse élevée de bibliothèques de composés permettant d'identifier des composés pharmaceutiques de pointe.
PCT/US2003/023075 2002-07-24 2003-07-24 Methodes destinees a identifier des petites molecules modulant la terminaison de traduction prematuree et la degradation de l'arnm induite par mutation non-sens Ceased WO2004010106A2 (fr)

Priority Applications (4)

Application Number Priority Date Filing Date Title
AU2003252136A AU2003252136A1 (en) 2002-07-24 2003-07-24 METHODS FOR IDENTIFYING SMALL MOLEDULES THAT MODULATE PREMATURE TRANSLATION TERMINATION AND NONSENSE MEDIATED mRNA DECAY
CA002493808A CA2493808A1 (fr) 2002-07-24 2003-07-24 Methodes destinees a identifier des petites molecules modulant la terminaison de traduction prematuree et la degradation de l'arnm induite par mutation non-sens
US10/521,775 US7927791B2 (en) 2002-07-24 2003-07-24 Methods for identifying small molecules that modulate premature translation termination and nonsense mediated mRNA decay
EP03765998A EP1543157A4 (fr) 2002-07-24 2003-07-24 Methodes destinees a identifier des petites molecules modulant la terminaison de traduction prematuree et la degradation de l'arnm induite par mutation non-sens

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US39834402P 2002-07-24 2002-07-24
US39833202P 2002-07-24 2002-07-24
US60/398,332 2002-07-24
US60/398,344 2002-07-24

Publications (2)

Publication Number Publication Date
WO2004010106A2 WO2004010106A2 (fr) 2004-01-29
WO2004010106A3 true WO2004010106A3 (fr) 2004-08-26

Family

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PCT/US2003/023075 Ceased WO2004010106A2 (fr) 2002-07-24 2003-07-24 Methodes destinees a identifier des petites molecules modulant la terminaison de traduction prematuree et la degradation de l'arnm induite par mutation non-sens

Country Status (5)

Country Link
US (1) US7927791B2 (fr)
EP (1) EP1543157A4 (fr)
AU (1) AU2003252136A1 (fr)
CA (1) CA2493808A1 (fr)
WO (1) WO2004010106A2 (fr)

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US8822663B2 (en) 2010-08-06 2014-09-02 Moderna Therapeutics, Inc. Engineered nucleic acids and methods of use thereof
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US9107886B2 (en) 2012-04-02 2015-08-18 Moderna Therapeutics, Inc. Modified polynucleotides encoding basic helix-loop-helix family member E41
US9283287B2 (en) 2012-04-02 2016-03-15 Moderna Therapeutics, Inc. Modified polynucleotides for the production of nuclear proteins
US9334328B2 (en) 2010-10-01 2016-05-10 Moderna Therapeutics, Inc. Modified nucleosides, nucleotides, and nucleic acids, and uses thereof
US9428535B2 (en) 2011-10-03 2016-08-30 Moderna Therapeutics, Inc. Modified nucleosides, nucleotides, and nucleic acids, and uses thereof
US9464124B2 (en) 2011-09-12 2016-10-11 Moderna Therapeutics, Inc. Engineered nucleic acids and methods of use thereof
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US20060257866A1 (en) 2006-11-16
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